A Study With Ruxolitinib Phosphate Cream Applied Topically to Subjects With Alopecia Areata (AA)

• Sponsor: Incyte Corporation (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02553330
• Collaborator: (none)
• Enrollment: 90
• Locations: 18
• Arms: 2
• Actual Duration (Months): 22.5
• Patients Per Site: 5
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

A phase 2 study to find out if the drug ruxolitinib Phosphate Cream is safe and has beneficial effects in people who have alopecia areata (partial or complete hair loss) when applied to the skin.

Condition or Disease	Intervention/Treatment	Phase
Alopecia Areata	Drug: Placebo Cream; Drug: Ruxolitinib Phosphate Cream	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 90 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: An Open-Label (Part A) and a Double-Blind, Randomized, Placebo Controlled (Part B) Study, With an Open-Label Extension, of Ruxolitinib Phosphate Cream Applied Topically to Subjects With Alopecia Areata
• Actual Study Start Date: Nov 18, 2015
• Actual Primary Completion Date: Oct 3, 2017
• Actual Study Completion Date: Oct 3, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ruxolitinib Phosphate Cream Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks; Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.	Drug: Ruxolitinib Phosphate Cream; Other Names: INCB018424
Placebo Comparator: Placebo Cream Part B: Double-blind treatment is 24 weeks (and/or treatment with Ruxolitinib Phosphate Cream if eligible) and follow-up is an additional 12 weeks.	Drug: Placebo Cream; Drug: Ruxolitinib Phosphate Cream; Other Names: INCB018424

Outcome Measures

Primary Outcome Measures

1. Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). [Up to Week 24]

   Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.

2. Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented) [Week 24]

   Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.

Secondary Outcome Measures

1. Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. [Up to Week 24]

   Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

2. Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). [Week 24]

   Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

3. Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. [Weeks 4, 8, 12, 18, and 24.]

   Number of participants achieving 90% or greater improvement in their SALT score (SALT90) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

4. Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3 [Baseline to Week 24]

   Number of subjects achieving a Physician's Global Assessment (PGA) score of 3 or above at week 24 (0, no regrowth; 1, <25% of regrowth; 2, 25%-49% of regrowth; 3, 50%-74% of regrowth; 4, 75%-99% of re- growth; 5, 100% of regrowth).

5. Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). [Weeks 4, 8, 12, and 18.]

   Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes

6. Part B: Mean Change From Baseline in SALT Score [Weeks 4, 8, 12, 18, and 24.]

   Severity of Alopecia Tool Score (SALT) calculation is based on a scoring system. The scalp is divided into the following 4 areas: 1) Vertex: 40% (0.4) of scalp surface area, 2) Right profile of scalp: 18% (0.18) of scalp surface area, 3) Left profile of scalp: 18% (0.18) of scalp surface area, and 4) Posterior aspect of scalp: 24% (0.24) of scalp surface area. The percentage of hair loss in any of these areas is the percentage hair loss multiplied by percent surface area of the scalp in that area. The SALT score is the sum of percentage of hair loss in all the above-mentioned areas, so a lower number indicates a better outcome. The reported SALT score range is from a minimum of 0 (no hair loss) to a maximum of 100 (100% hair loss).

7. Part A and B : Number of Treatment-emergent Adverse Events [Up to 100 weeks]

   Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Inclusion Criteria:

• Diagnosis of AA

• Duration and extent of current episode of AA

• Evidence of active hair loss

Exclusion Criteria:

• Evidence of diffuse, spontaneous terminal hair regrowth

• Receipt of treatment known to potentially affect the course of AA within last month

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Birmingham	Alabama	United States
2		Hot Springs	Arkansas	United States
3		New Haven	Connecticut	United States
4		Trumbull	Connecticut	United States
5		Miami	Florida	United States
6		Beverly	Massachusetts	United States
7		Boston	Massachusetts	United States
8		Clinton Township	Michigan	United States
9		Fort Gratiot	Michigan	United States
10		Fridley	Minnesota	United States
11		Minneapolis	Minnesota	United States
12		Winston-Salem	North Carolina	United States
13		Cleveland	Ohio	United States
14		Portland	Oregon	United States
15		Austin	Texas	United States
16		Houston	Texas	United States
17		Katy	Texas	United States
18		Norfolk	Virginia	United States

Sponsors and Collaborators

• Incyte Corporation

Investigators

• Study Director: Kathleen Butler, MD, Incyte Corporation

Study Documents (Full-Text)

• Study Protocol - Oct 12, 2016
• Statistical Analysis Plan - Oct 27, 2015

More Information

Publications

None provided.

• Responsible Party: Incyte Corporation
• ClinicalTrials.gov Identifier: NCT02553330
• Other Study ID Numbers: INCB 18424-204
• First Posted: Sep 17, 2015
• Last Update Posted: Dec 16, 2020
• Last Verified: Nov 1, 2020

• Keywords provided by Incyte Corporation: Hair loss; alopecia areata; alopecia totalis; alopecia universalis; JAK inhibitors; topical hair growth
• Additional relevant MeSH terms: Alopecia; Alopecia Areata

Study Results

Participant Flow

Recruitment Details	This study was conducted at 17 study centers in the United States.
Pre-assignment Detail	Twelve participants were enrolled at 4 sites and were evaluable in the Part A treatment period; In Part B, 78 additional participants were enrolled at 17 sites.

Arm/Group Title	Part A: Ruxolitinib Cream	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Period Title: Overall Study
STARTED	12	39	39
COMPLETED	7	0	2
NOT COMPLETED	5	39	37

Baseline Characteristics

Arm/Group Title	Part A: Ruxolitinib Cream	Part B : Placebo Cream	Part B : Ruxolitinib Cream	Total
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.	Total of all reporting groups
Overall Participants	12	39	39	90
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	47.6 (13.17)	44.3 (15.92)	42.4 (14.69)	43.4 (15.25)
Sex: Female, Male (Count of Participants)
Female	9 75%	24 61.5%	27 69.2%	60 66.7%
Male	3 25%	15 38.5%	12 30.8%	30 33.3%
Race/Ethnicity, Customized (Count of Participants)
White or Caucasian	11 91.7%	33 84.6%	29 74.4%	73 81.1%
Black or African American	0 0%	5 12.8%	4 10.3%	9 10%
Asian	1 8.3%	1 2.6%	0 0%	2 2.2%
Other	0 0%	0 0%	5 12.8%	5 5.6%
Unknown	0 0%	0 0%	1 2.6%	1 1.1%
Race/Ethnicity, Customized (Count of Participants)
Hispanic or Latino	1 8.3%	3 7.7%	5 12.8%	9 10%
Not Hispanic or Latino	11 91.7%	36 92.3%	33 84.6%	80 88.9%
Not Reported	0 0%	0 0%	1 2.6%	1 1.1%

Outcome Measures

1. Primary Outcome

Title	Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented).
Description	Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.
Time Frame	Up to Week 24

Outcome Measure Data

Analysis Population Description

Part A evaluable population in treatment period includes all subjects exposed to at least 1 dose of study drug in that period.

Arm/Group Title	Part A: Ruxolitinib Cream
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks
Measure Participants	12
Week 4	0 0%
Week 8	0 0%
Week 12	3 25%
Week 18	4 33.3%
Week 24	6 50%

2. Primary Outcome

Title	Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented)
Description	Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.
Time Frame	Week 24

Outcome Measure Data

Analysis Population Description

Intent to Treat population in the Part B treatment period i.e all participants who are randomized

Arm/Group Title	Part B : Placebo Cream	Part B: Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	39	39
Count of Participants [Participants]	5 41.7%	5 12.8%

3. Secondary Outcome

Title	Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair.
Description	Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time Frame	Up to Week 24

Outcome Measure Data

Analysis Population Description

The subgroup analysis was performed on subjects exposed to at least 1 dose of study drug in that period with a baseline SALT score of 50-100%.

Arm/Group Title	Part A: Ruxolitinib Cream
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks
Measure Participants	7
Week 4	0 0%
Week 8	0 0%
Week 12	2 16.7%
Week 18	3 25%
Week 24	4 33.3%

4. Secondary Outcome

Title	Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented).
Description	Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time Frame	Week 24

Outcome Measure Data

Analysis Population Description

The subgroup analysis was performed on subjects exposed to at least 1 dose of study drug in that period with a baseline SALT score of 50-100%.

Arm/Group Title	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	22	21
Count of Participants [Participants]	2 16.7%	4 10.3%

5. Secondary Outcome

Title	Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair.
Description	Number of participants achieving 90% or greater improvement in their SALT score (SALT90) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.
Time Frame	Weeks 4, 8, 12, 18, and 24.

Outcome Measure Data

Analysis Population Description

Part A evaluable population in treatment period includes all subjects exposed to at least 1 dose of study drug in that period. Intent to Treat population in the Part B treatment period i.e all participants who are randomized.

Arm/Group Title	Part A: Ruxolitinib Cream	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks.	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	12	39	39
Week4	0 0%	0 0%	0 0%
Week 8	0 0%	0 0%	0 0%
Week 12	0 0%	0 0%	1 2.6%
Week 18	1 8.3%	0 0%	2 5.1%
Week 24	2 16.7%	0 0%	2 5.1%

6. Secondary Outcome

Title	Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3
Description	Number of subjects achieving a Physician's Global Assessment (PGA) score of 3 or above at week 24 (0, no regrowth; 1, <25% of regrowth; 2, 25%-49% of regrowth; 3, 50%-74% of regrowth; 4, 75%-99% of re- growth; 5, 100% of regrowth).
Time Frame	Baseline to Week 24

Outcome Measure Data

Analysis Population Description

Intent to Treat population in the Part B treatment period i.e all participants who are randomized. Participants dropped out of study by week 24 were excluded from analysis.

Arm/Group Title	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks.	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	33	33
Count of Participants [Participants]	5 41.7%	2 5.1%

7. Secondary Outcome

Title	Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented).
Description	Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes
Time Frame	Weeks 4, 8, 12, and 18.

Outcome Measure Data

Analysis Population Description

Intent to Treat population in the Part B treatment period i.e all participants who are randomized.

Arm/Group Title	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks.	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	39	39
Week 4	0 0%	0 0%
Week 8	0 0%	2 5.1%
Week 12	1 8.3%	3 7.7%
Week 18	4 33.3%	5 12.8%

8. Secondary Outcome

Title	Part B: Mean Change From Baseline in SALT Score
Description	Severity of Alopecia Tool Score (SALT) calculation is based on a scoring system. The scalp is divided into the following 4 areas: 1) Vertex: 40% (0.4) of scalp surface area, 2) Right profile of scalp: 18% (0.18) of scalp surface area, 3) Left profile of scalp: 18% (0.18) of scalp surface area, and 4) Posterior aspect of scalp: 24% (0.24) of scalp surface area. The percentage of hair loss in any of these areas is the percentage hair loss multiplied by percent surface area of the scalp in that area. The SALT score is the sum of percentage of hair loss in all the above-mentioned areas, so a lower number indicates a better outcome. The reported SALT score range is from a minimum of 0 (no hair loss) to a maximum of 100 (100% hair loss).
Time Frame	Weeks 4, 8, 12, 18, and 24.

Outcome Measure Data

Analysis Population Description

Intent to Treat population in the Part B treatment period i.e all participants who are randomized.

Arm/Group Title	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks.	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	39	39
Change from Baseline to Week 4	1.02 (1.577)	0.13 (1.534)
Change from Baseline to Week 8	2.13 (2.233)	-1.11 (2.160)
Change from Baseline to Week 12	1.23 (2.956)	-2.55 (2.873)
Change from Baseline to Week 18	-0.01 (3.550)	-4.16 (3.461)
Change from Baseline to Week 24	-1.68 (3.886)	-3.92 (3.794)

9. Secondary Outcome

Title	Part A and B : Number of Treatment-emergent Adverse Events
Description	Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment
Time Frame	Up to 100 weeks

Outcome Measure Data

Analysis Population Description

All enrolled subjects who received at least 1 dose of INCB018424 or Placebo.

Arm/Group Title	Part A: Ruxolitinib Cream	Part B : Placebo Cream	Part B : Ruxolitinib Cream
Arm/Group Description	Part A: Open-label treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks	Part B: Double-blind treatment is 24 weeks and/or optional treatment with Ruxolitinib Phosphate Cream if eligible and follow-up is an additional 12 weeks.	Part B: Double-blind treatment is 24 weeks, an optional open-label treatment extension is 24 weeks if eligible, and follow-up is an additional 12 weeks.
Measure Participants	12	38	39
Treatment Period	9 75%	28 71.8%	23 59%
Extension Period	7 58.3%	23 59%	22 56.4%

Adverse Events

Time Frame	Up to 100 weeks
Adverse Event Reporting Description	The safety population included all randomized subjects who received at least 1 dose of study drug.
Arm/Group Title	Placebo Cream		Ruxolitinib Cream
Arm/Group Description	Part B: Double-blind treatment for 24 weeks.		Part A: Open-label treatment for 24 weeks. Part B: Double-blind treatment for 24 weeks. Placebo group from Part B had the option to crossover after the first 24 weeks of treatment period. Both Part A and Part B participants, if eligible, continued on to open-label extension (24 weeks) and long-term extension (48 weeks) periods.
All Cause Mortality
	Placebo Cream		Ruxolitinib Cream
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0%)		0/83 (0%)
Serious Adverse Events
	Placebo Cream		Ruxolitinib Cream
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0%)		4/83 (4.8%)
General disorders
Non-cardiac chest pain	0/38 (0%)	0	1/83 (1.2%)	1
Infections and infestations
Diverticulitis	0/38 (0%)	0	1/83 (1.2%)	1
Sepsis	0/38 (0%)	0	1/83 (1.2%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Infected neoplasm	0/38 (0%)	0	1/83 (1.2%)	1
Lentigo maligna	0/38 (0%)	0	1/83 (1.2%)	1
Psychiatric disorders
Generalised anxiety disorder	0/38 (0%)	0	1/83 (1.2%)	1
Other (Not Including Serious) Adverse Events
	Placebo Cream		Ruxolitinib Cream
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	14/38 (36.8%)		31/83 (37.3%)
Infections and infestations
Bronchitis	2/38 (5.3%)	2	2/83 (2.4%)	2
Nasopharyngitis	6/38 (15.8%)	6	13/83 (15.7%)	16
Sinusitis	1/38 (2.6%)	1	7/83 (8.4%)	7
Urinary tract infection	0/38 (0%)	0	5/83 (6%)	5
Skin and subcutaneous tissue disorders
Pruritus	3/38 (7.9%)	3	10/83 (12%)	11
Skin exfoliation	3/38 (7.9%)	3	3/83 (3.6%)	3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Clinical Study Agreement